FDA Grants Orphan Drug Designation to Cereno Scientific’s Lead Candidate CS1 for PAH

Joana Carvalho, PhD avatar

by Joana Carvalho, PhD |

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The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to CS1, Cereno Scientific’s lead therapy candidate for the treatment of pulmonary arterial hypertension (PAH).

CS1 is a unique reformulation of valproic acid, an anti-seizure medication that has been used for decades to treat epilepsy. The therapy works by blocking the enzyme histone deacetylase (HDAC), which is responsible for making chemical modifications that make DNA less accessible for the production of proteins.

By inhibiting HDAC, CS1 acts as an epigenetic modulator — molecules that control the activity of certain genes through chemical modifications — with strong anti-thrombotic (blood clots), anti-inflammatory, anti-fibrotic (tissue scarring), and pressure-relieving properties, that together make it a promising candidate to treat PAH.

“We are very pleased that the FDA has granted orphan drug designation to our HDAC inhibitor CS1, which allows us to implement a clinical development program on the rare and serious disease PAH aiming to help a broader group of patients, that now includes patients with rare diseases, with our lead compound CS1,” Sten R. Sörensen, CEO of Cereno Scientific, said in a press release.

“Our focus to develop drug candidates that effects epigenetic modulation, stems from our commitment at Cereno Scientific to help patients with cardiovascular related diseases,” Sörensen added.

Cereno submitted an application to the agency requesting the designation in December 2019.

Orphan drug status is given to support investigative treatments for rare diseases, defined in the U.S. as disorders that affect less than 200,000 people. The designation comes with certain benefits, including financial incentives for drug development and commercialization, U.S. market exclusivity for seven years following approval, FDA support in clinical trial design, fee exemptions and reductions.

Cereno also announced that, given the current COVID-19 pandemic, the “evaluation of epigenetic modulation for rare diseases is … not affected by the virus spread. The evaluation has moved into a more active phase since the company was granted Orphan Drug Designation for Pulmonary Arterial Hypertension.”

Preclinical studies on CS014 and CS036, two other HDAC inhibitors currently being developed by the company, are proceeding as planned.

However, Cereno’s future Phase 2 trial evaluating CS1, which was planned to start in mid-2020, has been postponed due to the pandemic.

The trial, which intends to evaluate CS1’s ability to prevent blood clots in patients undergoing elective orthopedic surgery, cannot be launched at this time because hospitals are lowering the priority for knee joint surgeries while focusing their resources on helping those infected with SARS-CoV-2. Traveling restrictions now in place to limit the spread of the pandemic also have contributed to the study’s delay.

The company has made arrangements to readjust the study’s launch to late 2020, but may make additional adjustments if necessary.

A Conversation With Rare Disease Advocates